PTC Therapeutics to present new Sephience data at SSIEM 2026

PTC Therapeutics will present new data on its phenylketonuria treatment Sephience at the 2026 SSIEM Annual Symposium in Helsinki, Finland, from 25 to 28 August. The presentations include results from clinical trials and real-world evidence showing sustained metabolic control, significant diet liberalisation and a consistent safety profile across the full spectrum of PKU patients, including those with classical PKU.

The company said the data demonstrate that treatment with Sephience led to a large number of responsive participants achieving normalisation of blood phenylalanine levels in a rapid timeframe. Sephience is approved in the US, EU, Japan and other countries for the treatment of hyperphenylalaninemia in patients one month of age and older with sepiapterin-responsive PKU.